Researchers at the University of Helsinki discovered that gene therapy stimulating blood vessel growth in the heart can alleviate cardiac atrophy caused by doxorubicin. The study found that this treatment prevented blood vessel rarefaction in mouse hearts, providing a potential solution for protecting patients from cardiotoxicity.
The Cell Press has partnered with The American Society of Gene and Cell Therapy (ASGCT) to publish its four journals, including Molecular Therapy, starting January 2017. This partnership aims to improve the dissemination of research breakthroughs in gene and cell therapies.
Researchers have successfully used gene therapy to correct the faulty NPC1 gene in mice with Niemann-Pick disease type C1, improving symptoms and lifespan. The treatment has the potential to halt disease progression and improve quality of life for patients.
A new human immunodeficiency has been discovered due to a faulty RASGRP1 gene, affecting T cells, B cells, and Natural Killer cells. The study identified a potential treatment using the drug lenalidomide, which reversed some effects of the mutation.
Researchers discovered a rare genetic variant in TM2D3 linked to late-onset Alzheimer's disease among Icelandic participants. The variant is estimated to increase the risk of Alzheimer's by approximately six times.
A portable device enables medical staff to genetically modify blood stem cells at a lower cost and with reduced staffing requirements. The technology has the potential to make gene therapy more accessible to patients in developing countries, reducing the need for expensive clean rooms.
A Phase 1 clinical trial demonstrated the long-term safety of gene transfer in treating neovascular age-related macular degeneration. The therapy produced therapeutic proteins encoded by genes for at least 2.5 years in patients with advanced NVAMD.
Blocking furin protein may offer unexpected benefits for patients with hemophilia A. Gene therapy and protein replacement treatments may be more effective with this approach.
Researchers at Binghamton University found that a genetic polymorphism in the CD40 gene modifies the severity of Duchenne muscular dystrophy, pointing to potential therapeutic approaches. The study suggests that drugs targeting CD40 may improve patient symptoms.
Researchers have prevented the development of Alzheimer's disease in mice by using a virus to deliver a specific gene, PGC-1 - alpha, into the brain. The study opens avenues for potential new treatments for the disease and suggests that injections of the gene may be beneficial in the early stages of the disease.
Researchers have engineered a novel, virus-like shell that can self-assemble and carry therapeutic DNA or RNA into human cells. This breakthrough could advance gene therapy treatments for rare genetic diseases and common conditions like diabetes.
Researchers developed a lentiviral vector-based gene therapy approach to deliver normal copies of the alpha-iduronidase gene to HSCs, indicating safety and efficacy in mice. The study sets the stage for a clinical trial to determine if MPS I patients can be successfully treated with this approach.
Researchers at Rosalind Franklin University and Oregon Health & Science University developed a breakthrough study on treating congenital diseases in utero using antisense oligonucleotides injected into the amniotic cavity. The procedure resulted in targeted alteration of gene expression for up to a month after birth.
Boston University researchers have identified a new strategy to treat colon cancers driven by the mutant KRAS gene. The study found that inhibiting MEK and TAK1 genes suppresses the growth of these cancer cells, providing a potential avenue for precision medicine.
Researchers developed a precision-engineered gene therapy virus that selectively silences BCL11A, reducing signs of sickle cell disease and increasing fetal hemoglobin production. The approach has the potential to substantially increase the ratio of non-sickling versus sickling hemoglobin.
Researchers found that CHD1-depleted human prostate cancer cells are hypersensitive to DNA breaks and chemotherapeutic drugs, including PARP inhibitors. This suggests CHD1 as a potential biomarker for targeted prostate cancer therapy.
Researchers at Penn University have found remarkable similarities between human Leber congenital amaurosis and canine blinding disease Senior Løken Syndrome. The diseases share the same causative gene, NPHP5, and display similar pathology. The study's findings offer promising results for developing therapies to treat these conditions.
The Generation Study will enroll over 1,300 cognitively healthy older adults at high risk of developing Alzheimer's due to inherited APOE gene mutations. Researchers will test two investigational treatments: an active immunotherapy and an oral medication to stop amyloid accumulation.
Researchers have developed a gene therapy approach using ultrasound energy and microbubbles to selectively open cells, allowing delivery of therapeutic agents. The study's findings will help refine this technique as a clinical tool and translate it into an effective gene or drug delivery tool for patients.
Researchers at UC Berkeley discovered a way to boost CRISPR-Cas9 cutting efficiency up to fivefold by disrupting DNA repair mechanisms with short oligonucleotide pieces. This technique increases the success rate of creating knockouts, essential for studying gene function and correcting hereditary mutations.
Researchers found that naturally infected chimpanzees developed antibodies able to neutralize multiple AAV serotypes, a hurdle for gene therapy. The study provides insights into the immune response to natural AAV infection and its implications for gene delivery strategies.
A new study analyzes breast tumors before and after hormone-reduction therapy, revealing the genetic complexity of these tumors and their varying responses to estrogen-deprivation treatments. The findings suggest that analyzing a single sample is insufficient for understanding how a patient should be treated.
Researchers at the National Institutes of Health have identified a novel genetic mutation that may lead to progressive loss of motor function in children. The study, published in Science Signaling, found that a gain-of-function mutation in the KCC3 protein causes extreme swelling of neurons, leading to nerve damage and muscle weakness.
The article explores the link between reward deficiency syndrome, dopamine dysregulation, and amino-acid therapy. Research suggests that individuals with certain gene variants may benefit from neuronutrient-amino-acid therapy to prevent addictive behaviors.
Researchers developed a hydrogel patch that delivers gene therapy, chemotherapy, and thermal ablation to treat colon cancer. The patch achieves complete tumor remission in non-resected tumors and prevents recurrence when applied after surgery.
The TAILOR-PCI Study aims to determine whether prescribing antiplatelet therapy based on a patient's CYP2C19 genotype can prevent heart attack, stroke, and cardiovascular death. The study, which began in 2013, plans to enroll 5,270 patients and investigate the effects of genomics on treatment outcomes.
A new gene therapy approach successfully delivered a therapeutic gene to the brains of treated mice, restored enzyme function, and extended survival. The treatment holds promise for developing similar therapies for humans and targeting additional brain disorders.
Researchers from the University of Pennsylvania have shown that early exposure to the human IDUA protein increased immune tolerance, allowing for widespread expression of the enzyme and resolution of brain lesions. The study informs the planning of first-in-human trials for MPS I treatment.
A new study found no serious adverse events related to gene therapy in patients with advanced Parkinson's disease five years after treatment. The long-term safety profile of the therapy is promising, lending support to its potential as a treatment for this debilitating disease.
A new gene therapy approach has shown promising results in treating hereditary tyrosinemia type 1 (HT1), a metabolic disorder characterized by progressive liver disease. The treatment involves transplanting corrected liver cells into the diseased liver, resulting in enzyme production and preventing liver failure.
Researchers have developed a gene delivery system that can be applied as an eye drop, rapidly moving from the surface to the retina and remaining for over eight hours. This method shows promise for treating several eye diseases, including diabetic retinopathy and age-related macular degeneration.
Researchers have identified three new risk genes for amyotrophic lateral sclerosis (ALS) using genetic data from Project MinE. One of these genes, C21orf2, increases the risk of developing ALS by 65%. The study's findings could aid the development of personalized treatments using gene therapy.
A recent study by Sanford Health identified gene mutations that could impact treatment for adult cancer patients. The study found nearly 90% of patients had genes that matched treatment options, with 40% able to receive personalized therapy.
Researchers have identified three new genes that increase the risk of motor neuron disease (MND), a debilitating condition with no effective treatments. The discovery provides new opportunities for targeted research and potentially improved outcomes for Australian patients, who are expected to benefit from increased genetic discoveries.
Researchers describe expanding scope of gene therapy targeting CNS diseases, including Alzheimer's and ALS. The article highlights rapid progress in viral vector development and delivery strategies for treating these conditions.
Australian researchers have developed a novel gene therapy that targets muscle cells to prevent cachexia, a condition of weight loss and frailty associated with severe muscle wasting. The approach has shown promise in pre-clinical mouse models, suggesting potential benefits for treating frailty in other chronic illnesses.
A new study discovered 172 novel cancer gene-drug combinations that selectively kill cancer cells without harming healthy cells. This breakthrough expands the number of actionable mutations and provides a promising approach to personalized cancer therapy.
A new study has developed a methodology to analyze bacterial virulence factors and identify therapeutic targets for developing effective antimicrobial agents. The TREP approach was applied to Haemophilus influenzae, a respiratory pathogen that causes COPD symptoms, revealing potential blocking elements for invasion.
Researchers have successfully programmed a virus to transport genetic material to affected tissue and nerve cells, opening up new options for treating various diseases. The approach uses attenuated viruses to selectively target diseased cells, reducing damage to healthy ones.
A study at the University of Texas MD Anderson Cancer Center found that genetic mutations in DNA mismatch repair deficiency (dMMR) are present in 15% of colorectal cancer cases. The study identified MSH2 and MSH6 as the most commonly affected genes, providing a new approach to diagnosis and treatment using precision medicine.
Researchers have developed a technique to program stem cells to grow new cartilage on a 3-D template shaped like the ball of a hip joint. The cartilage can release anti-inflammatory molecules to fend off arthritis. The discovery may provide an alternative to hip-replacement surgery, particularly in younger patients.
Researchers have found that chromatin remodeling proteins, essential for DNA replication and gene expression, are frequently mutated in human cancers. These mutations indicate tumor suppressive functions, making chromatin remodelers promising new targets for targeted cancer therapy.
A new study from TSRI researchers reveals the role of a gene called Mkx in maintaining and strengthening tendons in animal models. The findings may bring doctors closer to using gene therapies to grow and repair tendons.
Scientists at Northwestern University have identified a novel target for depression treatment by manipulating HCN channels in the hippocampus. Reducing these channel's function has been shown to improve mood in mice, offering new hope for millions of patients who don't respond to existing treatments.
A clinical isolate of bacterial pathogen carrying the colistin resistance gene mcr-1 has been detected in humans in the US, sparking concerns about antibiotic resistance. The isolate was found to be susceptible to several commonly used antimicrobial agents, but its plasmid-mediated nature is still unknown.
Researchers have developed a lung-targeted gene therapy strategy that may improve treatments for inherited diseases like emphysema. The new approach, which delivers a normal copy of the gene to lung cells, shows promise in reducing disease severity and increasing protein production.
Researchers developed an anti-apoE4 specific monoclonal antibody that reverses key brain-related pathological effects of apoE4 and associated cognitive deficits in mice. The treatment also accumulates in the brain and binds to apoE4, suggesting a novel approach for AD therapy.
Researchers identified a network of genes shared by CLL patients and associated with patient survival, which could be targets for treatment. Epigenetic analysis is expected to become widely used for selecting personalized treatments in cancer and other diseases.
Brendan Lee receives 2016 Curt Stern Award for his groundbreaking work on human inborn errors of metabolism and structural birth defects of the skeleton. The award recognizes his significant scientific contributions over the past decade, including identifying genetic causes of chrondrodysplasia and Marfan syndrome.
Researchers have identified a genetic mutation in the TEK gene that causes primary congenital glaucoma, a devastating eye disease affecting children worldwide. The finding validates previous research in mice and suggests a new therapeutic pathway to repair damaged vessels in the eye.
A University of Colorado Cancer Center study used whole-exome sequencing to characterize genetic alterations in 25 commonly used bladder cancer cell lines. The researchers found 76 altered genes, many involving oncogenes like TERT and TP53, which can predict patient response to cisplatin treatment
A new hybridization-induced aggregation technology detects KRAS mutations in lung and colorectal cancers, enabling targeted therapies. The technique is simple, rapid, and inexpensive, with 100% agreement with sequencing results.
Researchers develop new method to generate large pool of gene-corrected cells, which can repopulate in vivo and treat various disorders
Researchers have identified a protein called TST that helps detoxify waste products in fat cells, leading to improved insulin sensitivity and reduced weight gain in obese mice. The study's findings suggest a potential new approach to treating type 2 diabetes associated with obesity.
A team of scientists has identified the Sostdc1 gene as a key regulator of periosteum stem cells during fracture repair, which could lead to new therapeutic treatments for difficult-to-heal injuries. The study found that mutant mice lacking the gene had thicker, denser cortical bone that healed at an accelerated rate.
A new study discovered a genetic variant that reduces the risk of coronary heart disease in people taking antidiabetic medications, contradicting previous assumptions about these drugs' safety. The variant was linked to the glucagon-like peptide-1 receptor and may provide valuable insights for predicting drug side effects earlier in th...
Researchers have discovered a combination of cells and genes that can repair damaged heart tissues in animal models of myocardial infarction. The study found that different biological treatments target various aspects of cardiac function and scarring, with some showing significant improvements in contractile function and angiogenesis.
Recent evidence suggests that AAV vectors used for gene therapy can trigger liver cancer in rare patients. A new article challenges re-interpreted data, reaffirming the link between insertional mutagenesis and hepatocellular carcinoma.
Researchers have developed a novel therapeutic strategy for treating cystic fibrosis, restoring lung cell function to 50% of healthy levels. By using a yeast genetic model, they identified key targets that can rescue the misfolding of the deltaF508-CFTR protein, which affects nearly 90% of patients with cystic fibrosis.
A new gene therapy has been developed to repair lungs damaged by chronic allergic asthma, reducing inflammation and improving airway structure and function. The study used a novel biodegradable platform to deliver the thymulin gene therapy, showing effective results in mice with experimental asthma.